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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT03596957
Other study ID # PoCKET
Secondary ID
Status Recruiting
Phase Phase 4
First received
Last updated
Start date September 12, 2018
Est. completion date April 2020

Study information

Verified date November 2018
Source Nordsjaellands Hospital
Contact Lisbet Brandi, MD DMSc MHM
Phone +45 48295993
Email lisbet.brandi@regionh.dk
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

Investigator initiated controlled multi-centre trial in a Prospective, Randomised, Open, Blinded Endpoint (PROBE) design.

Patients will be randomised in a 1:1 ratio either to treatment with tolvaptan for six weeks followed by six weeks observation without trial medication or no tolvaptan treatment, but following the same visit and investigation plan as the subjects taking tolvaptan.


Description:

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic kidney disease and the fourth leading cause of end-stage renal disease in adults Worldwide.

The tolvaptan tablet has been approved by EMA (European Medicines Agency) with the indication of slowing the progression of cysts development and renal insufficiency in adults with ADPKD. It is the newest and only possible treatment for this patient group and could be initiated in patients with evidence for rapidly progressive disease Development.

There is however in Denmark and other countries both scientific and financial reluctance to initiate this expensive treatment for several reasons e.g. selection of patients who might benefit, effect on progression of kidney disease, side effects and tolerability.

Before deciding on implementation in Denmark, more knowledge is needed. The results of the PoCKET trial will contribute with guidance on this decision.

Foremost the trial is designed to address not only the change in kidney volume, but the change in kidney function, which is what matters to the patients and their prognosis in terms of postponing time to end stage renal disease. Furthermore, important data on side effects and tolerability will be generated.


Recruitment information / eligibility

Status Recruiting
Enrollment 90
Est. completion date April 2020
Est. primary completion date June 2019
Accepts healthy volunteers No
Gender All
Age group 18 Years to 65 Years
Eligibility Inclusion Criteria:

- Adult patients between 18 and 65 years

- Diagnosis of typical ADPKD

- tKV above or equal to 750 ml by MRI scanning

- Estimated GFR (e-GFR) by CKD-EPI formula of above or equal to 45 mL/min/1.73 m2

Exclusion Criteria:

- Kidney transplant recipient

- Known liver disease except for liver cysts relating to ADPKD

- ASAT and ALAT above upper normal level

- Current treatment with thiazide and thiazide-line diuretics, mineral corticoid receptor antagonists, amiloride or loop diuretics

- Evidence of urinary tract obstruction

- Current treatment with CYP3A4 inhibitors

- Active malignant disease

- Current or previous treatment with tolvaptan

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Tolvaptan
At baseline the tolvaptan dosing will start with daily morning and afternoon doses of 45 mg and 15 mg respectively, with weekly increases to 60 mg and 30 mg and then to 90 mg and 30 mg according to subject tolerability

Locations

Country Name City State
Denmark Rigshospitalet - Site 42 Copenhagen
Denmark Herlev Hospital Herlev
Denmark Nordsjaellands Hospital - Site 41 Hillerød
Denmark Odense University Hospital - Site 45 Odense Odense C
Denmark Sjællands University Hospital Roskilde Roskilde
Denmark Aarhus University Hospital - Site 43 Skejby Aarhus N

Sponsors (1)

Lead Sponsor Collaborator
Lisbet Brandi

Country where clinical trial is conducted

Denmark, 

Outcome

Type Measure Description Time frame Safety issue
Primary Change in total Kidney Volume (tKV) measured by MRI scanning The change in the total Kidney Volume after six and 12 weeks participation in the trial Between baseline and six weeks and between six and 12 weeks
Secondary Changes in GFR The changes in GFR measured by Cr-EDTA clearance Between baseline and six weeks and between baseline and 12 weeks
Secondary Changes in relevant genetic and non-genetic biomarkers associated with CKD and ESRD Prediction of change in progression of the disease over time in the genes PKD1, PKD2, PKHD1 and HNF1B. The following biomarkers will be determined: NGAL, UMOD, MCP-1, KIM-1, cystatin-C and copeptin Between baseline and six weeks and between baseline and 12 weeks
Secondary Changes in Quality of Life Questionnaire SF36 Health Survey - with 36 questions to subject's health and wellbeing Between baseline and six weeks and between baseline and 12 weeks
Secondary Subject estimation of own health Estimated by a Visual Analogue Scale from 0 (worth wellbeing) to 100 (best wellbeing Between baseline and six weeks and between baseline and 12 weeks
Secondary Changes in ASAT and ALAT Changes estimated from laboratory results Between baseline and six weeks and between baseline and 12 weeks
Secondary Incidence of Adverse Events Evaluation of Adverse Events including severity, causality, outcome and seriousness assessments Between baseline and six weeks and between baseline and 12 weeks
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